Springfield-based microbiology researcher Andrew Wilber, a director of cell biology and hematology at Southern Illinois University School of Medicine, was recently named SIU’s 2026 Inventor of the Year for his work on developing gene therapies for rare blood disorders such as sickle cell disease and beta thalassemia.
Wilber, who has a doctorate in molecular genetics, told Illinois Times his research helped lead to MiNiRoLu, a gene therapy drug for those with the aforementioned blood disorders.
“What we’re doing is we’re using the patient’s own cells so that every individual is their own donor, and I think that’s what makes it accessible,” he said. “Just like any (genetic) therapy treatment, the cost is still an issue but we are trying to keep things as lean as possible in order to be under that insurance cap that that exists for many different treatments.”
Beta thalassemia, a genetic disorder, causes an individual to produce less hemoglobin and red blood cells. Individuals with sickle cell disease suffer from a different gene defect causing misshapen red blood cells that die off early, resulting in chronic fatigue and pain. Those with sickle cell disease, which primarily afflicts Black people, have average lifespans more than 20 years shorter than those without the disorder, according to the American Society of Hematology.
Wilber noted that the patient population for those with blood disorders is often portrayed in incorrect lights.
“The other big issue, particularly (for) sickle cell patients, is that when they’re young, under 18 (years old), their care is quite good, in general. It’s once they hit 18, and now they’re considered an adult… they’re considered just drug-seeking, when they’re looking for medications to relieve that severe pain that they have. That’s one of the complications that exists with this patient population. The other one is the access to care is not the same across the board,” he said. “They don’t have a lot of options available to them, bone marrow transplant being the one real curative option, (but) even bone marrow transplants are limited.”
In December 2023, the Food and Drug Administration approved the first gene therapies to treat sickle cell disease. Those treatments cost between $2 million and $3 million per patient and require chemotherapy.
Wilber’s MiNiRoLu is still undergoing studies and trials with the National Institutes of Health, but has a pharmaceutical company dedicated to blood disorder, San Rocco Therapeutics, ready to produce the therapy.
“We’re working with our colleagues at the NIH in order to be able to demonstrate the utility of (the drug), to identify the dose of it that we need to use to be able to introduce this new gene into a sufficient number of cells to have a therapeutic benefit,” Wilber said. “These are just the standard hoops that you have to jump through in order to get to the point where not only are you confident that it can potentially work, but the regulatory body is also confident that it can potentially work and not cause harm is the main thing.
“We’re not the first ones to attempt this… we’ve learned from what others have already done and tried to make a product that we believe is going to, while not first in class, be best in class and hopefully to be able to be more affordable,” he said.